Ibrutinib Phase 2 Study Results in Patients with Mantle Cell Lymphoma Published in The New England Journal of Medicine

    Ibrutinib Phase 2 Study Results in Patients with Mantle Cell Lymphoma
               Published in The New England Journal of Medicine

PR Newswire

SUNNYVALE, Calif., June 19, 2013

SUNNYVALE, Calif., June 19, 2013 /PRNewswire/ -- Pharmacyclics, Inc. (the
"Company") (Nasdaq: PCYC) today announced that The New England Journal of
Medicine (NEJM) published results of a Phase 2 study evaluating the
investigational oral Bruton's tyrosine kinase (BTK) inhibitor ibrutinib in
patients with relapsed/refractory mantle cell lymphoma (MCL) online. These
data suggested ibrutinib may be effective and generally well-tolerated in
patients with relapsed/refractory MCL.

Results of a separate study examining the safety and efficacy of ibrutinib
monotherapy for the treatment of patients with relapsed/refractory chronic
lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), some of whom
had deletion of part of chromosome 17 (del 17p), has also been published
online by The NEJM. Pharmacyclics sponsored both studies and is jointly
developing ibrutinib with Janssen Research & Development, LLC.

"There remains a significant unmet need for patients with MCL," said lead
author Michael Wang, M.D., Department of Lymphoma/Myeloma, The University of
Texas MD Anderson Cancer Center. "These data are exciting because
theydemonstrate that the longer MCL patients are treated withibrutinib the
better they respond to the treatment."

The study evaluated 111 patients with relapsed/refractory MCL treated with
ibrutinib. The majority of patients (86 percent) had intermediate or high-risk
MCL and had previously undergone treatment with a median of three prior
therapiesbefore enrollment in the study. Patients were enrolled in two
cohorts based on whether they had prior exposure to bortezomib. Overall
response rate across both cohorts was 68 percent with 47 percent of
patientsachieving a partial response and 21 percent achieving a complete
response where all signs of cancer are gone. The estimated median response
duration was 17.5 months. The median progression free survival was 13.9 months
and while the median overall survival for this study has not yet been reached
it is estimated to be 58 percent at 18 months. The overall response to
ibrutinib was independent of prior treatment including bortezomib and
lenalidomide or underlying risk/prognosis, bulky disease, gender or age.In an
initial subset of patients enrolled in the study who have the longest follow
up (n=51), the response to ibrutinib increased with longer duration of
treatment, with the complete response rate increasing from 16 percent at a
median follow-up of 3.7 months to 37 percent at a median follow-up of 18.2

The majority of adverse events (AEs) were Grade 1 or 2 in severity, with the
most common AEs attributed to ibrutinib being diarrhea, infections and
fatigue. Only 7 percent of patients irrespective of relationship experienced
an AE leading to treatment discontinuation. Grade 3-4 hematological toxicities
were infrequent, with neutropenia (16 percent), thrombocytopenia (11 percent)
and anemia (10 percent) as the most frequent hematological AEs reported.

Data from this study were presented at the annual meeting of the American
Society of Hematology in December 2012, the European Hematology Association
annual meeting in June 2013 and are being presented at the 12th International
Conference on Malignant Lymphoma in Lugano, Switzerland this week.

Study Design
The Phase 2, open-label, multicenter study was designed to determine the
safety and efficacy of ibrutinib in patients with relapsed or refractory MCL.
The primary endpoint of the study was overall response rate. Secondary
endpoints included: duration of response, progression free survival, overall
survival and frequency and severity of adverse events.

The study enrolled patients with a confirmed diagnosis of relapsed or
refractory MCL at 18 sites internationally, and 86 percent of patients had
intermediate or high-risk MCL. Study participants received a 560mg daily oral
dose of ibrutinib monotherapy and weretreated into two cohorts based on prior
exposure to bortezomib – either no prior bortezomib (n=63) or prior bortezomib
(n=48). Overall, patients had received a median of three prior therapies.

About Mantle Cell Lymphoma
MCL, a B-cell malignancy, is an aggressive type of B-cell non-Hodgkin lymphoma
(NHL) that usually occurs in older adults. The disease typically begins in the
lymph nodes but can spread to other tissues, such as bone marrow and liver.
Ibrutinib targets the B-cell receptor pathway an important pathway in
malignant B-cell growth and proliferation. In the United States there are
approximately 5,000 new cases of MCL each year.

About Ibrutinib
Ibrutinib was designed to specifically target and selectively inhibit an
enzyme called Bruton tyrosine kinase (BTK). BTK is a key mediator of at least
three critical B-cell pro-survival mechanisms occurring in parallel
—regulation of apoptosis, cell adhesion, and cell migration and homing.

The effectiveness of ibrutinib alone or in combination with other treatments
is being studied in several B-cell malignancies, including chronic lymphocytic
leukemia/small lymphocytic lymphoma, mantle cell lymphoma, diffuse large
B-cell lymphoma, follicular lymphoma, Waldenstrom's macroglobulinemia and
multiple myeloma. To datesix Phase 3 trials have been initiated with
ibrutinib and a total of 30 trials are currently registered on
www.clinicaltrials.gov. Janssen Biotech, Inc. and Pharmacyclics entered a
collaboration and license agreement in December 2011 to co-develop and
co-commercialize ibrutinib.

About Pharmacyclics
Pharmacyclics^® is a clinical-stage biopharmaceutical company focused on
developing and commercializing innovative small-molecule drugs for the
treatment of cancer and immune mediated diseases. Our mission and goal is to
build a viable biopharmaceutical company that designs, develops and
commercializes novel therapies intended to improve quality of life, increase
duration of life and resolve serious unmet medical healthcare needs; and to
identify promising product candidates based on scientific development
expertise, develop our products in a rapid, cost-efficient manner and pursue
commercialization and/or development with partners when and where appropriate.

Presently, Pharmacyclics has two product candidates in clinical development
and several preclinical molecules in lead optimization. The Company is
committed to high standards of ethics, scientific rigor, and operational
efficiency as it moves each of these programs to viable commercialization.

The Company is headquartered in Sunnyvale, California and is listed on NASDAQ
under the symbol PCYC. To learn more about how Pharmacyclics advances science
to improve human healthcare visit us at http://www.pharmacyclics.com.

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Ramses Erdtmann
SVPof Investor Relations and Corporate Communications
Phone: 408-215-3325

U.S. Medical Information, Pharmacyclics:
855-ibrutinib [(855) 427-8846]

SOURCE Pharmacyclics, Inc.

Website: http://www.pharmacyclics.com
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